Cleveland Clinic Survey Shows Big Pharma Innovation Renaissance

Each year the Cleveland Clinic, one of the foremost academic medical centers in the United States, asks a panel of 110 of its physicians and scientists for their choices as to the most promising medical breakthroughs that will impact their patients in the coming year. What is great about the list is the immediacy that the prospective treatments will soon start to have on health care. Four of these innovations involve medical technologies such as painless blood testing for those afraid of needles, a wireless cardiac pacemaker that can be implanted in the heart without surgery, and single-dose radiation therapy for breast cancer.

However, six of the breakthroughs involve novel drugs designed to treat some of the world’s major killers: cancer, heart failure, and lung disease. What is also striking is that many of these breakthroughs are being driven by Big Pharma, whose critics often question its ability to innovate.

Take, for example, the field of immune-oncology, which is the hottest area of cancer research today. New drugs, known as PD-1 inhibitors, enable one’s immune system to attack tumors. Merck’s Keytruda has recently been approved for melanoma, but it has potential in other cancers as well with studies underway in lung and kidney cancer. Astra-Zeneca, Roche, and
Bristol-Myers Squibb also have drugs in late stage development thus ensuring multiple options for patients in need of these promising drugs.

The Cleveland Clinic’s panel also highlighted a second cancer approach, antibody-drug conjugates (ADCs). ADCs bind anti-cytotoxic drugs to synthetic antibodies which are designed to bind only to cancer cells. This results in safer and more effective treatment as healthy cells are spared from the drugs’ cell killing properties. Roche’s Kadcyla and Adcertris from
Seattle Genetics are now on the market and, again, a number of others are in late stage development.

Heart disease is also an area of pharmaceutical innovation.
Novartis recently announced late stage clinical results for LCZ696, which combines valsartan with sacubitril, an angiotensin-receptor neprilysin inhibitor. When directly compared to standard treatment in heart failure patients, LCZ696 reduced the risk of cardiovascular death by 20%. This drug has been given fast-track status by the FDA, welcome news to the 5 million U.S. patients currently with heart failure. There is intense competition in another class of drugs to treat heart disease, antibodies to a protein known as PCSK-9. These injectable drugs potently lower LDL-cholesterol and will benefit those for whom statins are not effective enough. The leaders are
Sanofi/Regeneron,
Amgen, and Pfizer.

Idiopathic pulmonary fibrosis (IPF) is a life-threatening disease that scars the lung, leading to breathing difficulties that reduce oxygen delivery to the brain and elsewhere. Until recently, there were few treatment options for IPF patients. However, the FDA recently approved Intermune’s pirfenidone and Boehringer Ingelheim’s nintedanib, drugs that significantly slow IPF progression greatly enhancing the quality of life for these patients.

Finally, and perhaps most significantly, the Cleveland Clinic’s panel recognized Sanofi’s vaccine for Dengue Fever. This is a deadly tropical disease that affects millions of people in economically depressed regions. Sanofi plans to file for global approvals early in 2015 and hopefully this vaccine will be available for those at risk by the end of 2015.

By no means has Big Pharma been solely responsible for these major advances. The biotech industry has also played a role. But the names that dominate read like a Big Pharma’s “Who’s who”: Merck, Novartis, Roche, Sanofi, Pfizer, AstraZeneca, Bristol-Myers Squibb, and Boehringer Ingelheim. While pharma-scolds attack the industry for a lack of innovation or for focusing on life-style drugs, the Cleveland Clinic’s survey instead teaches that Big Pharma is driving progress in the major causes of death in the world. And for those who think that Big Pharma only works on drugs for those who can afford them, Sanofi’s decades long effort in finding a vaccine for Dengue Fever is a wonderful example of the industry’s philanthropic R&D efforts.

I was the president of Pfizer Global Research and Development in 2007 where I managed more than 13,000 scientists and professionals in the United States, Europe, and Asia. I've received numerous awards including an Honorary Doctor of Science degree from the University of Ne...